A new study has brought scientists one step further in the direction of developing a cure for a brutal group of rare brain ...

Until recently, even the most advanced gene therapies could only be given after a child was born—often racing against time to prevent irreversible damage. In the first part of this series, we explored ...

Following up on previous, dimly received issuances, a new set of ideas published by the FDA to streamline regulatory pathways ...

As newborn screening and rapid DNA sequencing become routine, we are poised to catch and treat inherited diseases at their earliest stages. Today, we can intervene in the first days or weeks of life.

A CRISPR-Cas9-based therapy targeting the gene encoding transthyretin greatly reduced transthyretin levels in patients with ...

Stanford researchers and their collaborators have revealed a new device that could change the way scientists conduct ...

Myrtelle Inc. ("Myrtelle" or the "Company"), a gene therapy company pioneering transformative treatments for ...

Alison Clare has received funding from the Wellcome Trust and the Medical Research Council. Her position is currently funded by National Institute for Health and Care Research (NIHR) Biomedical ...

DMD gene therapy faces challenges in safety, efficacy, and long-term outcomes, despite FDA approval of delandistrogene moxeparvovec. Future strategies focus on novel AAV capsids, dual/triple vector ...

New Enterprise Associates led the investment round, with contributions from newcomer Eli Lilly & Company, among others.

Dr. Singh underscored India’s remarkable progress in the biotechnology and pharmaceutical sectors, calling it a key driver of ...